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uniQure Announces First Clinical Data From Second Dose Cohort of AMT-060 in Ongoing Phase I/II trial in Patients with Severe Hemophilia B | ||
By: Nasdaq / GlobeNewswire - 03 Dec 2016 | Back to overview list |
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-- Second-dose Cohort Demonstrates Dose Response with All Patients Free of Prophylactic FIX Replacement Therapy and Only One Spontaneous Bleed Reported -- Low-Dose Cohort Shows Sustained Levels of FIX Activity Up To One Year, with Marked Reduction of Bleeding Over Time and a Complete Cessation of Spontaneous Bleedings in Last 14 Weeks of Observation --No Activation of T-Cell Responses or Loss of FIX Activity in Any Patients --Investor Webcast Monday, December 5, 2016 at 7:00 a.m. PST/10 a.m. EST Lexington, MA and Amsterdam, the Netherlands, December 4, 2016 - uniQure N.V. (Nasdaq: QURE), a leader in human gene therapy, today announced new and updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B. The data includes up to 52 weeks of follow-up from the low-dose cohort and up to 31 weeks of follow-up from the second dose cohort. New data presented from the second-dose cohort show a dose response with substantial improvement in disease state in all five patients, including the discontinuation of precautionary Factor IX (FIX) infusions in all four patients that previously required chronic replacement therapy. To date, only one spontaneous bleed was reported after discontinuation of prophylactic FIX replacement therapy. All five patients in the low-dose cohort, who bleedings were previously uncontrolled despite being managed with prophylactic therapy, continue to maintain robust, constant and clinically meaningful levels of FIX activity for up to 52 weeks post treatment, with a complete cessation of spontaneous bleedings in the last 14 weeks of observation.
These clinical data from both patient cohorts were presented last evening in a poster session of the 58th American Society of Hematology (ASH) Annual Meeting taking place in San Diego, California.
"In total, we are observing a therapeutic benefit from AMT-060 that is clearly superior to their previous prophylactic FIX replacement therapy regimen, even in patients with advanced joint disease who still experienced many bleeds despite prophylaxis with FIX," he added. "The safety and clinical efficacy profile observed in this study, together with the higher FIX expression levels support selection of the 2x10 13 gc/kg dose for a pivotal registration trial."
Phase 1/2 Trial Overview
"The data from our Phase I/II study demonstrate AMT-060 is delivering sustained and significantly improved clinical benefits to patients suffering from severe hemophilia B by enabling them to discontinue frequent infusions of FIX replacement therapy and to essentially eliminate the risk of spontaneous bleeding," stated Christian Meyer, M.D., Ph.D., chief medical officer at uniQure. "Importantly, none of the patients treated with AMT-060 have lost FIX activity for up to one year post administration. To date, our insect-cell manufactured AAV5 gene therapies have been administered to 22 patients across three clinical studies without any evidence of AAV5 capsid-specific cellular immune responses or long-term safety complications." "The proprietary elements of AMT-060, including our fully-humanized FIX gene cassette and AAV5 vector, are the only gene therapy components clinically demonstrated in hemophilia B to be safe, effective, and durable for up to six and a half years," he added. "We believe these factors, along with our commercial-scale manufacturing capabilities, differentiate AMT-060 from other hemophilia gene therapies in development, and we look forward to advancing our program into a late-stage clinical study."
AMT-060 is being co-developed with Chiesi for Europe.
Date and Time:
Monday, December 5, 2016 at 7:00 am PST/ 10:00 am EST
To request attendance at the meeting, please RSVP to
Investors@uniQure.com
as space is limited
Hemophilia B is a serious and rare inherited disease in males characterized by insufficient blood clotting. The condition can lead to repeated and sometimes life-threatening episodes of external and internal bleeding following accidental trauma or medical interventions. Severe hemophilia is characterized by recurrent episodes of spontaneous joint bleeds, that cause long-term damage to the joints resulting in disabling arthropathy. Bleeds may be fatal if they occur in the brain. The deficient blood clotting results from the lack of functional human Factor IX, or hFIX. Treatment of hemophilia B today consists of prophylactic or on-demand protein replacement therapy, in which one to three times weekly intravenous administrations of plasma-derived or recombinant hFIX are required to prevent bleeding and once daily infusions in case bleeding occurs. Hemophilia B occurs in approximately 1 out of 30,000 live births. About uniQure uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with CNS, liver/metabolic and cardiovascular diseases. www.uniQure.com uniQure Forward-Looking Statement
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release.
These forward-looking statements include, but are not limited to, statements regarding the development of our gene therapy product candidates, including the future development of AMT-060, the success of our collaborations and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our product candidates. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators' clinical development activities, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's 2015 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 4, 2016. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.
Maria E.Cantor
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This announcement is distributed by NASDAQ OMX Corporate Solutions on behalf of NASDAQ OMX Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein. Source: uniQure via GlobeNewswire
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